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Cystinosis Summary

Description: A metabolic disease characterized by the defective transport of CYSTINE across the lysosomal membrane due to mutation of a membrane protein cystinosin. This results in cystine accumulation and crystallization in the cells causing widespread tissue damage. In the KIDNEY, nephropathic cystinosis is a common cause of RENAL FANCONI SYNDROME.

Also Known As: Cystinoses

Networked: 497 relevant articles (13 outcomes, 42 trials/studies) for this Disease

Key Drugs and Agents for Cystinosis

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. Cysteamine (Mercaptamine) : 11 outcomes 16 studies in 133 results : FDA 1
  2. Cystine (L-Cystine) : 3 outcomes 18 studies in 257 results : IBA
  3. Nephropathic cystinosis : 1 outcome 3 studies in 22 results : IBA
  4. Vitamin D : 1 outcome in 5 results : FDA 10 Generic
  5. artemisinine (artemisinin) : 1 outcome in 2 results : IBA
  6. Electrolytes : 1 outcome in 1 result : IBA
  7. Cystamine : 1 outcome in 1 result : IBA
  8. Cysteine (L-Cysteine) : 2 studies in 14 results : FDA 10
  9. Glutathione (Reduced Glutathione) : 2 studies in 8 results : IBA
  10. Ophthalmic Solutions (Eye Drops) : 2 studies in 5 results : IBA
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Diseases Related to Cystinosis

  1. Fanconi Syndrome (Syndrome, Fanconi)
  2. Disease Progression
  3. Oculocerebrorenal Syndrome (Lowe Syndrome)
  4. Glycogen Storage Disease (Glycogenosis)
  5. Lysosomal Storage Diseases (Lysosomal Storage Disease)
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Key Therapies for Cystinosis

Efficacy Chart >>
  1. Kidney Transplantation : 1 outcome 1 study in 37 results
  2. Transplants (Transplant) : 1 study in 18 results
  3. Transplantation (Transplant Recipients) : 1 study in 15 results
  4. Homologous Transplantation (Allograft) : 1 study in 9 results
  5. Renal Replacement Therapy (Therapies, Renal Replacement) : 4 results
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